Introduction 1 2 3 4 5 6 7 8 Currently, no drugs have been approved by the Food and Drug Administration (FDA) for the preventive treatment of pediatric migraine. Topiramate is an antiepileptic drug, which has been approved by the FDA in the United States and in many other countries for the preventive treatment of migraine in adults and for the treatment of partial-onset seizures and primary generalized tonic–clonic seizures as add-on therapy in children as young as 2 years. Our aim was to review the medical literature concerning the efficacy and tolerability of topiramate in the prophylactic treatment of migraine in children and adolescents. Materials and methods 1 9 Table 1 AAN classification of evidence for therapeutic intervention Class I: Prospective, randomized, controlled clinical trial with masked outcome assessment, in a representative population. The following are required:  (a) Primary outcome(s) is/are clearly defined  (b) Exclusion/inclusion criteria are clearly defined  (c) Adequate accounting for drop-outs and crossovers with numbers sufficiently low to have minimal potential for bias  (d) Relevant baseline characteristics are presented and substantially equivalent among treatment groups or there is appropriate statistical adjustment for differences Class II: Prospective matched group cohort study in a representative population with masked outcome assessment that meets (a)–(d) above OR a RCT in a representative population that lacks one criteria (a)–(d). Class III: All other controlled trials (including well-defined natural history controls or patients serving as own controls) in a representative population, where outcome is independently assessed, or independently derived by objective outcome measurement. Class IV: Evidence from uncontrolled studies, case series, case reports, or expert opinion. Results We found two RCTs, a post-hoc subset analysis of 51 adolescents (12–17 years) who had been enrolled in three pivotal RCTs of topiramate for migraine prophylaxis in adults and two open studies. 10 11 P P P P 12 P P P P n n n 10 13 P P P 14 P 15 Unfortunately, the studies we reviewed were not homogeneous with regard to inclusion and exclusion criteria and with regard to outcome measures such as the “responder rate,” which was not considered in all studies. Furthermore, the degree of disability caused by the migraine episodes, which is essential in the decision to initiate prophylactic treatment, was not assessed in all studies. Discussion 16 10 12 Taken together, data from the papers we reviewed suggest that topiramate is effective in the preventive treatment of pediatric migraine. Furthermore, topiramate dosages, which seem to be effective in the treatment of migraine in children and adolescents (2–3 mg/kg/day) are much lower than those indicated for the adjunctive treatment of epilepsy (5–9 mg/kg/day) in children as young as 2 years. 10 12 10 10 14 In conclusion, topiramate seems to be a promising therapeutic option, though, clearly, further controlled trials are needed to confirm this data, as are studies comparing different drugs.